Krishnamurti Lakshmanan
Division of Hematology/Oncology/BMT Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center Health System, Pittsburgh, Pennsylvania 15213, USA.
Pediatr Hematol Oncol. 2007 Dec;24(8):569-75. doi: 10.1080/08880010701640531.
Sickle cell disease is associated with considerable morbidity and premature mortality. Hematopoietic cell transplantation offers the possibility of cure and is associated with excellent results in pediatric patients receiving stem cell transplantation from a matched sibling donor. Reduced intensity conditioning regimen have the potential to further reduce regimen related morbidity and mortality. Improved understanding of the natural history of complications such as stroke and pulmonary hypertension, effects of treatments, such as hydroxyurea and blood transfusions, as well as the impact of transplantation on organ damage are likely to influence the timing and indication of transplantation. Improvements in preparative regimen may enable the safe use of alternate source of stem cells such as unrelated matched donors and further improve the applicability and acceptability of this treatment.
镰状细胞病与相当高的发病率和过早死亡率相关。造血细胞移植提供了治愈的可能性,并且对于接受来自匹配同胞供体的干细胞移植的儿科患者而言,其效果极佳。降低强度的预处理方案有可能进一步降低与方案相关的发病率和死亡率。对诸如中风和肺动脉高压等并发症的自然病史、羟基脲和输血等治疗的效果以及移植对器官损伤的影响有更深入的了解,可能会影响移植的时机和指征。预处理方案的改进可能使诸如不相关匹配供体等替代干细胞来源得以安全使用,并进一步提高这种治疗的适用性和可接受性。